Biogen said the Food and Drug Administration had agreed to review its application for approval of a treatment for amyotrophic lateral sclerosis that failed to slow the progression of the disease in a Phase 3 trial.
Biogen (ticker: BIIB) is seeking approval of the drug, known as tofersen, for a rare, fatal, genetic form of ALS known as SOD1-ALS, which the company says affects only 330 people in the U.S. Last fall, Biogen shares dropped 4.1% in a single day after the company disclosed disappointing results of the Phase 3 trial of the drug.
At the time, Biogen said that while tofersen hadn't had a statistically significant effect on patients' clinical function, the "totality of evidence" showed that people who started using it earlier had "better outcomes." The company also said that it saw "trends favoring tofersen" in a number of secondary measures tracked in the trial, and in data on the drug's biological activity.
In June, Biogen presented new data from an extension of the trial that it said showed that earlier initiation of tofersen did slow the decline of patients compared with people who began using it later.
Biogen said early Tuesday that it was asking the FDA to approve tofersen through the agency's accelerated approval pathway, a mechanism designed to allow early access to drugs whose benefit for patients isn't yet proven. Often used to give the go-ahead to cancer treatments, it is the same pathway through which the FDA approved Biogen's Alzheimer's drug Aduhelm last summer.
The Aduhelm approval turned out to be extraordinarily controversial for the agency, leading to the resignation of three members of an outside FDA advisory panel that had ruled against the drug's efficacy. Despite the approval, the Center for Medicare and Medicaid Services determined this year that the Medicare program will effectively not cover Aduhelm.
Biogen is still reeling from the CMS decision. The company effectively ended its efforts to commercialize Aduhelm in May, and is seeking a new CEO. The company is facing a number of challenges, including new competition for its multiple sclerosis drug Tecfidera.
Investors are finding it hard to see the silver lining in the stock. Shares are down 50% since June 10 of last year, shortly after the FDA approved Aduhelm.
The news that the FDA will consider approval of tofersen didn't seem to immediately change the narrative. Biogen shares were up 0.5% in premarket trading on Tuesday, while shares of Ionis Pharmaceuticals (IONS), from which Biogen licensed tofersen in 2018, gained 2.2%.
"The available data show that tofersen has the potential to make a meaningful difference for people with SOD1-ALS," Biogen's interim head of research and development, Dr. Priya Singhal, said in a statement. "Pursuing the FDA's accelerated approval pathway offers the potential to make tofersen available to people living with this fatal, neurodegenerative disease as quickly as possible."
The company is seeking accelerated approval of the drug based on its ability to reduce proteins known as neurofilaments, which the company says predicts a slowing in the decline of patients.
The FDA will make a decision on whether to approve tofersen by Jan. 25. It will convene an advisory committee for an open public hearing to discuss the application.
For now, Biogen investors remain focused on Phase 3 data on lecanemab, another Alzheimer's drug that Biogen is developing with the Japanese pharmaceutical firm Eisai (ESALY). That data is expected this fall.
"We remain cautious as to whether tofersen's data should rise to a level that supports approval, and either way, the potential contribution to BIIB's long-term revenue would be far too immaterial to offset the looming lecanemab readout," RBC Capital Markets analyst Brian Abrahams wrote in a note out early Tuesday.
The FDA decision on tofersen might be more important as an indicator as to how flexible the FDA will be with accelerated approvals of neurological drugs.